Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births.

HT-100 is an orally available small molecule drug candidate being developed to reduce fibrosis and inflammation and to promote healthy muscle fiber regeneration in Duchenne patients. The application of HT-100 to Duchenne and other fibrotic diseases is based on pioneering work by Dr. Mark Pines at the Volcani Institute in Israel. Akashi, led by CEO Marc Blaustein, has been granted orphan designation for Duchenne in both the U.S. and EU, as well as Fast Track designation in the U.S. A phase 1b/2a clinical program is currently underway at five hospitals across the U.S.

The grant will be used to evaluate the safety, tolerability and pharmacokinetics of HT-100 in patients with Duchenne in both a phase 1b open-label, single and multiple ascending dose study and a phase 2a extension study.

Preclinical studies in young mice that lack dystrophin demonstrated that HT-100 reduced the amount of fibrosis that accumulates in the heart and diaphragm muscles and improved the ability of the mice to cling to a rotating rod. In older mice with established fibrosis in the diaphragm muscle the compound was able to reverse fibrosis and improve respiratory and cardiac function.

According to PPMD's Founding President and CEO, Pat Furlong, "Marc Blaustein and the team at Akashi are innovators and leaders in the Duchenne space. We are thrilled to be able to support this next critical step in the journey of HT-100, a therapy that we believe in. PPMD is excited to partner with Akashi and appreciate that, like us, they believe it is as important for a drug to be safe, as it is to be effective."

"PPMD was an early supporter of our efforts to develop HT-100 and this funding—following a thorough scientific review—comes at a crucial time as we continue to generate both safety and efficacy data from the HT-100 clinical program in boys with DMD," said Akashi CEO Marc Blaustein. "We are grateful for PPMD's continued financial support and pleased to build on our already-strong relationship with PPMD and the broader DMD patient community."

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 3,500-5,000 live male births (about 20,000 new cases worldwide each year). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures.

Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. Young men with Duchenne typically live into their late twenties.

Duchenne can be passed from parent to child, but approximately 35 percent of cases occur because of a random spontaneous mutation. In other words, it can affect anyone. Although there are medical treatments that may help slow its progression, there is currently no cure for Duchenne.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

About Akashi Therapeutics

Akashi Therapeutics is a clinical stage biopharmaceutical company whose mission is to develop treatments for Duchenne muscular dystrophy and other rare pediatric diseases. Akashi was founded by leading patient organizations and biotechnology industry veterans and is managed by a seasoned team of drug development experts to impact a central problem in rare diseases: rapid therapy development. Akashi is developing a pipeline of therapies with the goal of transforming Duchenne from a 100% fatal, aggressive muscle-wasting disease to a chronic, manageable condition. For more information, please visit www.akashirx.com.